Von Hippel-Lindau disease is a rare genetic disorder characterized by multisystem involvement. In this inherited disease, benign tumors and visceral cysts (fluid-filled sacs) are formed in certain parts of the body including the eyes, brain, spinal cord, and inner ears, among others. The incidence of von Hippel-Lindau disease in the general population is reported to be between 1 case per 27,000 and 1 case per 43,000 live births. The von Hippel-Lindau disease pipeline analysis by Expert Market Research focuses on various treatment options developing for the disease. The drugs are primarily focused on managing symptoms and surgically removing tumors. However, there is growing emphasis on the development of targeted therapies that go beyond symptom management and eliminate the need for invasive procedures.
Major companies involved in the von Hippel-Lindau disease pipeline analysis include Betta Pharmaceuticals Co., Ltd. and Peloton Therapeutics, Inc., among others.
Leading drugs currently under the pipeline include Belzutifan and oral HS-10516, among others.
Advancements in genetic research and robust regulatory support are significantly contributing to the expansion of the pipeline of investigational drugs for Von Hippel-Lindau disease.
The Von Hippel-Lindau Disease Pipeline Analysis Report by Expert Market Research gives comprehensive insights into von Hippel-Lindau disease drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for von Hippel-Lindau disease. The report includes the analysis of over 100 pipeline drugs and 50+ companies. The von Hippel-Lindau disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from von Hippel-Lindau disease.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to von Hippel-Lindau disease.
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Von Hippel-Lindau disease is an autosomal dominant condition in which tumors and cysts are formed in different parts of the body. These growths can be benign or cancerous. The disease is caused by mutations in the VHL tumor suppressor gene. Hemangioblastomas (in the brain and spinal cord), renal cell carcinoma (in the kidneys), and pheochromocytomas (in the adrenal glands), among others, include the common tumors associated with von Hippel-Lindau disease.
Surgery is commonly used to remove tumors that become malignant or cause significant issues. Advances in precision medicine and the growing focus on targeted approaches based on specific molecular pathways are anticipated to positively influence the drug pipeline landscape. Moreover, new drugs targeting HIF-2α and other pathways linked to von Hippel-Lindau disease are likely to improve patient outcomes in the coming years. In November 2024, Merck’s Welireg® (belzutifan) received approval from China’s National Medical Products Administration for treating adult patients with von Hippel-Lindau disease-associated tumors. This first-in-class HIF-2α inhibitor addresses renal cell carcinoma, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors, marking Merck’s 17th global approval and expanding treatment options in oncology.
Von Hippel Lindau incidence is estimated to be between 1 case per 27,000 and 1 case per 43,000 live births in the population. The prevalence rate is between 1 in 31,000 to 1 in 91,000 individuals. The mean diagnostic age is 26 years, with men and women getting affected equally. The majority of cases are diagnosed through a germline mutation.
This section of the report covers the analysis of von Hippel-Lindau disease drug candidates based on several segmentations including:
By Phase
The pipeline report covers 50+ drug analyses based on phase:
By Drug Class
The von Hippel-Lindau disease pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR von Hippel-Lindau disease pipeline analysis, phase II covers a major share of the total clinical trials for von Hippel-Lindau disease. Phase II constitutes the major share in the disease market drug pipeline at 57.1%, followed by Phase I at 42.9%. Meanwhile, Early Phase I, Phase III, and Phase IV currently hold no shares, offering promising opportunities for future development.
The drug molecule categories covered under the Von Hippel-Lindau Disease pipeline analysis include hypoxia-inducible factor (HIF) inhibitors, mTOR inhibitors, and VEGF pathway inhibitors. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for von Hippel-Lindau disease.
Small molecule inhibitors targeting hypoxia-inducible factor (HIF) are gaining prominence in the oncology market. For instance, HS-10516, a small molecule drug that inhibits HIF-2α, is under development for VHL syndrome associated tumors in China. After obtaining an exclusive license in May 2022, its capsules received NMPA clinical trial approval in June 2024 for VHL syndrome-related tumors.
The EMR report for the von Hippel-Lindau disease pipeline analysis covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Von Hippel-Lindau disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for vitiligo. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of von Hippel-Lindau disease drug candidates.
Sponsored by Peloton Therapeutics, Inc. (a subsidiary of Merck & Co., Inc.), the objective of this open-label Phase 2 clinical trial is to investigate the efficacy and safety of orally administered belzutifan treatment in an estimated 50 participants with von Hippel-Lindau disease with at least 1 measurable renal cell carcinoma tumor.
Jiangsu Hansoh Pharmaceutical Co., Ltd. is conducting a Phase I multi-center clinical trial to investigate the antitumor efficacy, safety, and pharmacokinetics of HS-10516, in around 60 Chinese subjects with von Hippel-Lindau disease syndrome-associated tumors.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Von Hippel-Lindau Disease Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Von Hippel-Lindau disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within Von Hippel-Lindau disease pipeline insights.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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